Access to antineoplastic drugs in LATAM & ESMO-MCBS

-A tool to separate chaff from the wheat in the era

of high cost drugs?

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Felipe RoitbergMedical Oncologist – Thoracic and Head & Neck Cancer Division- University of São Paulo (ICESP)- Hospital Sírio Libanês

Disclosure / Possible Conflicts of Interest

- Clinical Trials: Boehringer Ingelheim, Bristol Meyers Squibb, AstraZeneca, Roche

‒ Speaker/Lectures: Boehringer-Ingelheim, AstraZeneca, Bayer, Bristol MeyersSquibb, Merck Sharp Dohme

‒ Advisory Board: Boehringer Ingelhein

‒ No stocks from pharmaceutical companies

‒ ESMO, IALSC, SBOC member, State of São Paulo HTA affiliated, JGO reviewer

Cancer Drugs Costs

Source: Quinteles IMS and Reuters

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Perspectives

Survival Gain/CureQuality of Life/Toxicity

Survival Gain/CureQuality of Life

Hazard Ratio, RR, PFSStatistically significant

Cost-Effectiveness (QALY, DALY, ICER)Budget Impact

“Statistically Significant Benefit”

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OS Gain: 6,24m x 5,91 10 days

IC 95% HR 0,82 (0,69-0,99)

7J Clin Oncol 2015; 33:1191-1196

OS Benefit in 5years: 18,2 x 8,8%

Cytotoxics Miscellaneous Hormones

Bleomycin Doxorubicin Oxaliplatin Calcium folinate Anastrozole

Capecitabine Etoposide Paclitaxel Filgrastim Bicalutamide

Carboplatin Fluorouracil Procarbazine Imatinib Dexamethasone

Cisplatin Gemcitabine Vinblastine Trastuzumab Leuprorelin

Cyclophosphamide Ifosfamide + mesna Vincristine Zoledronic acid Tamoxifen

Dactinomycin Irinotecan Vinorelbine

Docetaxel Methotrexate

WHO Model List of Essential Medicines 20th List (March 2017) (Amended August 2017)

AA

EML Essential Medicines List– WHO (2015)

ESMO-MCBS

Quality of Life

Magnitude of Clinically

Benefit

Overall survival,

Progression free

survivaL

ToxicityPrognosis

of the condition

HR,Long term survival,

RR

Cherny et al, Ann Oncol 2015 & 2017

Factors taken into account for ESMO-MCBS Substantial improvements:A & B in curative and 5 & 4 in non curativesetting

Available at: https://www.esmo.org

Courtesy: Prof E. de Vries

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Evaluation form 1: for new approaches to adjuvant therapy or new potentially curative

therapiesGrade A

Mark with X if relevant

>5% improvement of survival at ≥3 years follow-upImprovements in DFS alone (primary endpoint) (HR <0.65) in studies without mature survival data

≥ 3% but ≤ 5% improvement at ≥3 years follow-upImprovement in DFS alone (primary endpoint) (HR 0.65 - 0.8) without mature survival dataNon-inferior OS or DFS with reduced treatment toxicity or improved Quality of Life (with validated scales)Non-inferior OS or DFS with reduced treatment cost as reported study outcome (with equivalent outcomes and risks)

<3% improvement of survival at ≥ 3 years follow-upImprovement in DFS alone (primary endpoint) (HR >0.8) in studies without mature survival dataImprovements in pCR alone (primary endpoint) by >30% relative AND >15% absolute gain in studies without mature survival data

Grade B

Grade C

Courtesy: Prof E. de Vries

Non Curative Therapies: more variables to address

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4

3

2

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Overall Survival

(Absolute Gain & HR)

Progression Free Survival

Others than OS & PFS

Quality Outcomes:• Toxicity• QOL

Single Arms Studies (Rare Diseases & Unmet Medical Needs)

“Every System is perfectly Designed to get the results it gets”

What Is Value in Health Care? Michael E. PorterN Eng J Med 363;26